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简要综述:修复破碎的心:生物治疗学的发展历程

Concise Review: Mending a Broken Heart: The Evolution of Biological Therapeutics.

作者信息

Chen Caressa, Termglinchan Vittavat, Karakikes Ioannis

机构信息

Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, California, USA.

Department of Cardiothoracic Surgery, Stanford University School of Medicine, Stanford, California, USA.

出版信息

Stem Cells. 2017 May;35(5):1131-1140. doi: 10.1002/stem.2602. Epub 2017 Mar 16.

Abstract

Heart failure (HF), a common sequela of cardiovascular diseases, remains a staggering clinical problem, associated with high rates of morbidity and mortality worldwide. Advances in pharmacological, interventional, and operative management have improved patient care, but these interventions are insufficient to halt the progression of HF, particularly the end-stage irreversible loss of functional cardiomyocytes. Innovative therapies that could prevent HF progression and improve the function of the failing heart are urgently needed. Following successful preclinical studies, two main strategies have emerged as potential solutions: cardiac gene therapy and cardiac regeneration through stem and precursor cell transplantation. Many potential gene- and cell-based therapies have entered into clinical studies, intending to ameliorate cardiac dysfunction in patients with advanced HF. In this review, we focus on the recent advances in cell- and gene-based therapies in the context of cardiovascular disease, emphasizing the most advanced therapies. The principles and mechanisms of action of gene and cell therapies for HF are discussed along with the limitations of current approaches. Finally, we highlight the emerging technologies that hold promise to revolutionize the biological therapies for cardiovascular diseases. Stem Cells 2017;35:1131-1140.

摘要

心力衰竭(HF)是心血管疾病常见的后遗症,仍然是一个严重的临床问题,在全球范围内与高发病率和死亡率相关。药理学、介入治疗和手术管理方面的进展改善了患者护理,但这些干预措施不足以阻止HF的进展,特别是终末期功能性心肌细胞不可逆转的丧失。迫切需要能够预防HF进展并改善衰竭心脏功能的创新疗法。在成功的临床前研究之后,出现了两种主要策略作为潜在解决方案:心脏基因治疗和通过干细胞及前体细胞移植进行心脏再生。许多潜在的基于基因和细胞的疗法已进入临床研究,旨在改善晚期HF患者的心脏功能障碍。在本综述中,我们关注心血管疾病背景下基于细胞和基因疗法的最新进展,重点介绍最先进的疗法。讨论了HF基因和细胞疗法的原理和作用机制以及当前方法的局限性。最后,我们强调了有望彻底改变心血管疾病生物疗法的新兴技术。《干细胞》2017年;35卷:1131 - 1140页

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/75c6/7069059/7037ea16da94/nihms853935f1.jpg

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